MX2021011757A - Polynucleotides, compositions, and methods for polypeptide expression. - Google Patents

Polynucleotides, compositions, and methods for polypeptide expression.

Info

Publication number
MX2021011757A
MX2021011757A MX2021011757A MX2021011757A MX2021011757A MX 2021011757 A MX2021011757 A MX 2021011757A MX 2021011757 A MX2021011757 A MX 2021011757A MX 2021011757 A MX2021011757 A MX 2021011757A MX 2021011757 A MX2021011757 A MX 2021011757A
Authority
MX
Mexico
Prior art keywords
compositions
methods
polynucleotides
polypeptide expression
benefits
Prior art date
Application number
MX2021011757A
Other languages
Spanish (es)
Inventor
Bradley Andrew Murray
Seth C Alexander
Christian Dombrowski
Original Assignee
Intellia Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of MX2021011757A publication Critical patent/MX2021011757A/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C12P21/02—Preparation of peptides or proteins having a known sequence of two or more amino acids, e.g. glutathione
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

Compositions and methods for gene editing. In some embodiments, a polynucleotide encoding Cas9 is provided that can provide one or more of improved editing efficiency, reduced immunogenicity, or other benefits.
MX2021011757A 2019-03-28 2020-03-27 Polynucleotides, compositions, and methods for polypeptide expression. MX2021011757A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962825656P 2019-03-28 2019-03-28
PCT/US2020/025372 WO2020198641A2 (en) 2019-03-28 2020-03-27 Polynucleotides, compositions, and methods for polypeptide expression

Publications (1)

Publication Number Publication Date
MX2021011757A true MX2021011757A (en) 2021-12-10

Family

ID=70416544

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021011757A MX2021011757A (en) 2019-03-28 2020-03-27 Polynucleotides, compositions, and methods for polypeptide expression.

Country Status (16)

Country Link
US (1) US20230012687A1 (en)
EP (1) EP3947670A2 (en)
JP (1) JP2022527302A (en)
KR (1) KR20220004649A (en)
CN (1) CN113993994A (en)
AU (1) AU2020248470A1 (en)
BR (1) BR112021019224A2 (en)
CA (1) CA3135172A1 (en)
CO (1) CO2021014400A2 (en)
EA (1) EA202192637A1 (en)
IL (1) IL286579A (en)
MA (1) MA55527A (en)
MX (1) MX2021011757A (en)
SG (1) SG11202110135YA (en)
TW (1) TW202102529A (en)
WO (1) WO2020198641A2 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
WO2023081689A2 (en) 2021-11-03 2023-05-11 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
WO2023133525A1 (en) * 2022-01-07 2023-07-13 Precision Biosciences, Inc. Optimized polynucleotides for protein expression
WO2023154749A2 (en) * 2022-02-09 2023-08-17 The Regents Of The University Of California In vitro and in vivo protein translation via in situ circularized rnas
WO2024044697A2 (en) * 2022-08-24 2024-02-29 Walking Fish Therapeutics, Inc. Compositions and methods for treatment of fabry disease

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
DK0618925T4 (en) 1991-12-24 2012-07-09 Isis Pharmaceuticals Inc Antisense oligonucleotides
JPH10500310A (en) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ PNA probes for the detection of Neisseria gonorrhoeae and Chlamydia trachomatis
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
EA015925B1 (en) * 2006-06-29 2011-12-30 ДСМ АйПи АССЕТС Б.В. A method for producing polypeptides
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
ES2741951T3 (en) 2012-12-17 2020-02-12 Harvard College Genetic engineering modification of the human genome guided by RNA
CA2904184C (en) 2013-03-08 2021-09-07 Novartis Ag Lipids and lipid compositions for the delivery of active agents
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
ES2774968T3 (en) 2013-12-19 2020-07-23 Novartis Ag Lipids and lipid compositions for the administration of active agents
EP4223285A3 (en) 2014-07-16 2023-11-22 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
KR20230156800A (en) 2015-03-03 2023-11-14 더 제너럴 하스피탈 코포레이션 Engineered crispr-cas9 nucleases with altered pam specificity
CN116590285A (en) 2015-09-21 2023-08-15 垂林克生物技术有限公司 Compositions and methods for synthesizing 5' -capped RNA
WO2017127750A1 (en) * 2016-01-22 2017-07-27 Modernatx, Inc. Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof
JP7245651B2 (en) 2016-03-30 2023-03-24 インテリア セラピューティクス，インコーポレイテッド Lipid Nanoparticle Formulations for CRISPR/CAS Components
EP3516056A1 (en) * 2016-09-23 2019-07-31 DSM IP Assets B.V. A guide-rna expression system for a host cell
WO2018067447A1 (en) 2016-10-03 2018-04-12 Itellia Therapeutics, Inc. Improved methods for identifying double strand break sites
EA202090873A1 (en) * 2017-09-29 2020-08-17 Интеллиа Терапьютикс, Инк. POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR EDITING THE GENOME

Also Published As

Publication number Publication date
EP3947670A2 (en) 2022-02-09
IL286579A (en) 2021-10-31
TW202102529A (en) 2021-01-16
MA55527A (en) 2022-02-09
JP2022527302A (en) 2022-06-01
US20230012687A1 (en) 2023-01-19
CA3135172A1 (en) 2020-10-01
WO2020198641A3 (en) 2020-11-05
CO2021014400A2 (en) 2021-11-19
AU2020248470A1 (en) 2021-11-11
WO2020198641A2 (en) 2020-10-01
EA202192637A1 (en) 2022-03-18
CN113993994A (en) 2022-01-28
KR20220004649A (en) 2022-01-11
BR112021019224A2 (en) 2021-11-30
SG11202110135YA (en) 2021-10-28

Similar Documents

Publication Publication Date Title
PH12020550359A1 (en) Polynucleotides, compositions, and methods for genome editing
MX2021011757A (en) Polynucleotides, compositions, and methods for polypeptide expression.
MX2019011040A (en) Compositions and methods for enhanced gene expression.
WO2018081535A3 (en) Dynamic genome engineering
WO2019089884A3 (en) Methods, compositions and components for crispr-cas9 editing of tgfbr2 in t cells for immunotherapy
MX2020004043A (en) Compositions and methods for gene editing for hemophilia a.
MX2018008266A (en) Cyclic dinucleotides for treating conditions associated with sting activity such as cancer.
MX2022002461A (en) Compositions and methods for cd123 modification.
MX2019004156A (en) TCRa HOMING ENDONUCLEASE VARIANTS.
MX2019014100A (en) Cblb endonuclease variants, compositions, and methods of use.
MX2022002462A (en) Compositions and methods for cll1 modification.
MX2021003457A (en) Compositions and methods for lactate dehydrogenase (ldha) gene editing.
MX2022003516A (en) Deoxyribonuclease uses in detergent composition.
MY195583A (en) Modified Homoserine Dehydrogenase and Method for Producing Homoserine Or L-Amino Acid Derived from Homoserine using the same
MX2020007591A (en) Modified homoserine dehydrogenase, and method for producing homoserine or homoserine-derived l-amino acid using same.
MX2019011272A (en) Compositions and methods for immunooncology.
MX2020006901A (en) Unnatural base pair compositions and methods of use.
MX2020014248A (en) Compositions and methods for genomic editing by insertion of donor polynucleotides.
MX2022002843A (en) Compositions and methods for modifying a plant characteristic without modifying the plant genome.
MX2021014528A (en) Methods and compositions for generating dominant alleles using genome editing.
MX2021000323A (en) Engineered pantothenate kinase variant enzymes.
MX2022000425A (en) Methods and compositions comprising reduced level of host cell proteins.
MX2022015284A (en) Compositions and methods for epigenome editing.
MX2021015657A (en) Oral care compositions comprising tin ions.
MX2018012872A (en) Compositions and methods for enhanced gene expression of pklr.